Tuesday, August 9, 2011

Results of Type 1 Diabetes Drug Trial

Q&A with Nicole Sherry, MD

Nicole Sherry, MD, director of the MassGeneral for Children Diabetes Center, explains the results of the first, large-scale immune therapy drug trial for type 1 diabetes. Dr. Sherry was the principal investigator at Massachusetts General Hospital of a multi-center study* of 513 patients, ages 8-35 undergoing therapy with teplizumab, a drug that the trial’s leaders hoped would help preserve the patient’s own beta cells.

The body’s beta cells are located in the pancreas and make insulin, a hormone that regulates the blood’s sugar levels. Though people with type 1 diabetes are born with a full supply of beta cells, at some point in their lives their bodies begin to attack the beta cells, compromising the regulation of blood sugar levels.

Michael Staveley-O’Carroll

Read about Michael Staveley-O’Carroll (right), a patient who participated in the teplizumab drug trial led by endocrinologist Nicole Sherry, MD (left).

Q: What was the goal of the study?

A: Most patients with type 1 diabetes go through a short period after diagnosis when they require less insulin to keep their sugar levels steady. This is what we call the “honeymoon phase.” The honeymoon phase varies by age— there tends to be a faster progression if you’re younger. If you’re younger it could be three to six months and if you’re older the period during which your own body is making a significant amount of insulin could be closer to a year.

Teplizumab was put into a phase three trial to see if it would help prolong the honeymoon phase for children and adults, or basically to slow down the destruction of the beta cells.

Q: What did you find?

A: We confirmed that it does have efficacy. The interesting thing is that when you do a phase three trial you have to set up one main hurdle—in this case it was that at one year patients would meet a certain measure of blood sugar control and a certain lower-than-expected amount of insulin use. It didn’t meet that one endpoint, but this is a new field and this is really the first phase three trial for immune therapy in type 1 diabetes and that endpoint had never before been tested.

I think the results were encouraging. I think the treatment should be able to move forward after another trial and maybe five years from now be on market. I think it’s a great step forward to show that there’s a drug that has a decent safety profile that could make an impact on affecting the body’s process of destroying the body’s beta cells, because we don’t have anything available now that can do this.

Q: Was there a difference in the effect of the drug on children versus adults?

A: We did also find an increased effect in children, which speaks to how the disease affects people in different ways. One thing that I think is really important is that a lot of drugs are not tested in children and this study has shown that it’s very important to test new drugs in children because drugs may show a different effect in children than in adults, especially in type 1 diabetes.

Q: Who is a candidate for this drug?

A: I think the candidates will be people of all ages with recent onset type 1 diabetes. There is also a trial studying the use of this drug (teplizumab) for prevention in people at risk for type 1 diabetes.

Q: What’s next for this treatment? Are there plans for long-term studies?

A: There will hopefully be another phase three study building on what was learned in this study.  If it happens we will have the trial at MassGeneral Hospital for Children when it opens.

*Sherry N, Hagopian W, Ludvigsson J, Jain SM, Wahlen J, Ferry RJ Jr, Bode B, Aronoff S, Holland C, Carlin D, King KL, Wilder RL, Pillemer S, Bonvini E, Johnson S, Stein KE, Koenig S, Herold KC, Daifotis AG; Protégé Trial Investigators. Teplizumab for treatment of type 1 diabetes (Protégé study): 1-year results from a randomised, placebo-controlled trial. Lancet. 2011 Aug 6;378(9790):487-97. Epub 2011 Jun 28.

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